TrialStudy of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
Trial details
- Registry ID
- NCT01247207
- Phase
- Phase 3
PTCT
PTC Therapeutics
Company identity has not yet been verified
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16Trials in this database
15Recorded changes · last 12 months
Partial coverageOnly the trials and history collected so far
Research sample: selected versions from May 2023 onward plus a preceding baseline where available. Only selected fields were retained; endpoint measures were truncated to 300 characters. This is not complete trial or sponsor history. Daily snapshots begin when collection starts.
Completion dates · Pro
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Included trials
Check company identity sourceLong-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome
Trial details
- Registry ID
- NCT02352896
- Phase
- Phase 2
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
Trial details
- Registry ID
- NCT03179631
- Phase
- Phase 3
A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
Trial details
- Registry ID
- NCT04336826
- Phase
- Phase 2
A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
Trial details
- Registry ID
- NCT04378075
- Phase
- Phase 2, Phase 3
A Study to Evaluate Efficacy and Safety of PTC299 (Emvododstat) in Hospitalized Participants With Coronavirus (COVID-19)
Trial details
- Registry ID
- NCT04439071
- Phase
- Phase 2, Phase 3
A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
Trial details
- Registry ID
- NCT04577352
- Phase
- Phase 2, Phase 3
A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants
Trial details
- Registry ID
- NCT04903288
- Phase
- Phase 2
A Study of PTC923 in Participants With Phenylketonuria
Trial details
- Registry ID
- NCT05099640
- Phase
- Phase 3
A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
Trial details
- Registry ID
- NCT05166161
- Phase
- Phase 3
A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
Trial details
- Registry ID
- NCT05218655
- Phase
- Phase 3
A Study of Unesbulin in Participants With Advanced Leiomyosarcoma (LMS)
Trial details
- Registry ID
- NCT05269355
- Phase
- Phase 2, Phase 3
Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS
Trial details
- Registry ID
- NCT05349721
- Phase
- Phase 2
A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
Trial details
- Registry ID
- NCT05485987
- Phase
- Phase 2
A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
Trial details
- Registry ID
- NCT05515536
- Phase
- Phase 3
A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
Trial details
- Registry ID
- NCT06302348
- Phase
- Phase 3
Latest recorded trial changes
A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
NCT05166161 · PTC Therapeutics
Participant count type
Recorded edit− Previous value
Estimated
+ Updated value
Actual
Enrollment · participant count
Recorded edit− Previous value
count: 200; type: ESTIMATED
+ Updated value
count: 247; type: ACTUAL
Completion date type
Recorded edit− Previous value
Estimated
+ Updated value
Actual
Primary completion date
Recorded edit− Previous value
2026-06-30
+ Updated value
2026-07-21
Recruitment status
Recorded edit− Previous value
Active not recruiting
+ Updated value
Completed
A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
NCT04577352 · PTC Therapeutics
Primary outcomes · main study measures
Text edit · unverified significance− Previous value
measure: Change From Baseline in the Modified Friedreich Ataxia Rating Scale (mFARS) Score at Week 72
+ Updated value
measure: Change From Baseline in the mFARS Score at Week 72 - Modified Intent-to-treat (mITT) Analysis Set; measure: Change From Baseline in the mFARS Score at Week 72 - Intent-to-treat (ITT) Analysis Set
The saved outcome text may be shortened. Check the source for full wording.
A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
NCT04378075 · PTC Therapeutics
Primary outcomes · main study measures
Text edit · unverified significance− Previous value
measure: Percent Change From Baseline to Week 24 in the Number of Observable Motor Seizures per 28 Days
+ Updated value
measure: Percent Change From Baseline to Week 24 in the Number of Observable Motor Seizures Per 28 Days During the Double-blind Period
The saved outcome text may be shortened. Check the source for full wording.
Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
NCT01247207 · PTC Therapeutics
Completion date type
Recorded edit− Previous value
Estimated
+ Updated value
Actual
Primary completion date
Recorded edit− Previous value
2025-04-30
+ Updated value
2026-02-10
Recruitment status
Recorded edit− Previous value
Enrolling by invitation
+ Updated value
Completed
A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
NCT05166161 · PTC Therapeutics
Primary completion date
Recorded edit− Previous value
2026-04-30
+ Updated value
2026-06-30
Recruitment status
Recorded edit− Previous value
Recruiting
+ Updated value
Active not recruiting
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
NCT03179631 · PTC Therapeutics
Primary outcomes · main study measures
Text edit · unverified significance− Previous value
measure: Slope of Change in 6-Minute Walk Distance (6MWD) Over 72 Weeks
+ Updated value
measure: DB Period: Change From Baseline in 6-Minute Walk Distance (6MWD) at Week 72 - Modified Intention-to-treat (mITT) Population; measure: DB Period: Change From Baseline in 6MWD at Week 72 - Intent-to-Treat (ITT) Population; measure: DB Period: Average Rate of Change From Baseline in 6MWD at Week 72 - mITT Population; measure: DB Period: Average Rate of Change From Baseline in 6MWD at Week 72 - ITT Population
The saved outcome text may be shortened. Check the source for full wording.
A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
NCT05218655 · PTC Therapeutics
Enrollment · participant count
Recorded edit− Previous value
count: 102; type: ACTUAL
+ Updated value
count: 101; type: ACTUAL
A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
NCT05218655 · PTC Therapeutics
Primary outcomes · main study measures
Text edit · unverified significance− Previous value
measure: Number of Participants With Adverse Events (AEs)
+ Updated value
measure: Number of Participants With Treatment-emergent Adverse Events (TEAEs)
The saved outcome text may be shortened. Check the source for full wording.